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Syncona Ltd on Wednesday said its portfolio company Freeline Therapeutics Holdings PLC has received positive initial safety, tolerability and enzyme activity data from its FLT201 trial for the treatment of Gaucher disease. The London-based investor in healthcare companies said the first two patients treated showed a ‘robust increase and normalisation’ of GCase enzyme levels and activity, which it said is an ‘important indication of the candidate’s therapeutic potential’. It added that there were no adverse effects as of 13 weeks following dosing in patient one, and for six weeks in patient two. It said these adverse effects resolved without intervention. A third patient has been scheduled for dosing. Chief Executive Chris Hollowood said: ‘We are very pleased to see this positive data from Freeline which demonstrates a compelling safety profile and robust enzyme activity for its gene therapy programme for the treatment of Gaucher disease. ‘This data strengthens our belief that FLT201 has the potential to be first and best-in-class gene therapy for Gaucher disease, a debilitating condition where there is a clear need for better treatment options’ Separately, the firm said another of its portfolio companies, SwanBio Therapeutics, has commenced the second dose-escalation cohort of its clinical trial of SBT101. It said the trial is assessing the safety and efficacy of its SBT101 treatment as a therapy for rare spinal condition adrenomyeloneuropathy. The firm noted that dosing for its low-dose cohort was completed in August and has been well-tolerated so far. As a result, it said it has successfully dosed the first patient in its high-dose cohort. Syncona said it will provide a further £8.2 million to SwanBio in order to carry out its dosing of the high-dose cohort. Following this, it said its investment in Syncona will amount to £77.1 million. Chief Executive Chris Hollowood commented: ‘Following a review of the patient data from the first cohort, we are encouraged that the DSMB has recommended the initiation of the higher-dose cohort and we look forward to seeing the data generated from this cohort. Our further investment of $10m will enable the company to deliver additional data to deliver its long-term potential.’ Syncona shares rose 0.9% to 122.08 pence each on Wednesday afternoon in London. Copyright 2023 Alliance News Ltd. All Rights Reserved.
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